We’re thrilled to announce that Dr. Shiming Chen has been awarded a Seed grant from the Center of Regenerative Medicine for her groundbreaking project titled “hiPSC-induced retinal organoids modeling CRX-linked retinopathies.” This pioneering research aims to establish three-dimensional retinal organoids—essentially human retinas in a dish—to model inherited retinal diseases (IRDs) linked to the cone-rod-homeobox (CRX) transcription factor.
Shiming Chen, PhD
Dr. Bernard and Janet R. Becker Distinguished Professor in Ophthalmology
- Email: chenshiming@nospam.wustl.edu
- Chen Lab page
- Research Profiles
- Director of the Molecular Genetics Core
The Project’s Core Objectives
Dr. Chen’s project is poised to revolutionize our understanding of CRX-linked retinopathies. The primary objectives include:
- Creating 3D Retinal Organoids: By using induced pluripotent stem cells (iPSCs) derived from patients with CRX mutations, the team will create retinal organoids to replicate the human retina’s structure and function in vitro.
- Comparative Analysis: These retinal organoids will be compared to normal or isogenic control organoids to observe the differences in photoreceptor differentiation. This comparison is critical to understanding how CRX mutations impact retinal development and lead to disease.
- Gene Therapy Testing: The project will also test the efficacy of adeno-associated virus (AAV)-mediated CRX gene therapy. This approach aims to correct the mutant phenotypes observed in the retinal organoids, potentially paving the way for new treatments.
Collaborations and Methodologies
This ambitious project brings together a multidisciplinary team, combining the expertise of the Chen Lab with key partnerships:
- hCTO Core at the Center of Regenerative Medicine: Providing essential support and resources for the project’s success.
- Dr. Chao Zhou in Biomedical Engineering: Contributing advanced techniques and engineering insights to refine the retinal organoid models.
The collaborative effort focuses on utilizing iPSCs from families affected by CRX-linked retinopathies. By meticulously establishing and analyzing these retinal organoids, the team seeks to unravel the complexities of disease pathogenesis and explore innovative treatment strategies.
Impact and Future Directions
The potential impact of this research is profound. By modeling CRX-linked retinopathies in a controlled laboratory setting, Dr. Chen’s team will gain invaluable insights into the molecular and cellular mechanisms driving these conditions. Moreover, successful demonstration of AAV-mediated CRX gene therapy in ameliorating mutant phenotypes could herald a new era of targeted treatments for not only CRX-linked diseases but other IRDs as well.
This project exemplifies the transformative power of regenerative medicine and cutting-edge biotechnology. As the research progresses, we anticipate significant advancements in translational research, bringing hope to patients and families affected by inherited retinal diseases.
Stay tuned for updates on this exciting journey towards pioneering treatments and unlocking the mysteries of retinal disease pathogenesis.